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身高转变:性类固醇如何影响马凡综合征的最终身高

Transforming tallness: how sex steroids influence final height in Marfan syndrome.

作者信息

Yamaguchi Adriana Banhos Carneiro, Siviero-Miachon Adriana Aparecida, Okuda Paola Matiko Martins, Oliveira E Silva Lívia Cristina, Bustamante Talita de Faria, Spinola-Castro Angela Maria

机构信息

Universidade Federal de São Paulo (UNIFESP), Escola Paulista de Medicina (EPM), Setor de Endocrinologia Pediátrica, São Paulo, SP, Brazil.

Universidade Federal de São Paulo (UNIFESP), Escola Paulista de Medicina (EPM), Setor de Endocrinologia Pediátrica, São Paulo, SP, Brazil; Hospital do Grupo de Apoio ao Adolescente e à Criança com Câncer (GRAACC), São Paulo, SP, Brazil.

出版信息

J Pediatr (Rio J). 2025 May-Jun;101(3):424-429. doi: 10.1016/j.jped.2025.01.007. Epub 2025 Mar 13.

Abstract

OBJECTIVE

To evaluate the effects of hormonal treatment with sex steroids on the final height of patients with tall stature, including those diagnosed with Marfan Syndrome (MS), over 15 years in an outpatient setting.

METHODS

This retrospective cohort study reviewed the medical records of patients referred for tall stature. Descriptive statistics characterized the samples, while independent and paired t-tests assessed changes in final height (FH) and height at the start of treatment (HTS). One-way analysis of variance (ANOVA) evaluated the impact of chronological age at the initiation of therapy, bone age at the start of treatment, and pubertal stage on FH and HTS.

RESULTS

A total of 55 individuals with tall stature (51 % male) were included, among whom 35 (64 %) had clinically confirmed MS. Of these, 34 (62 %) received low-dose steroid treatment. Patients treated during pre-puberty exhibited an average height increase of 25.56 cm (95 %CI 20.40-30.73; p < 0.001; d = 2.86), while those treated during puberty showed an average gain of 11.93 cm (95 %CI 8.69-15.18; p < 0.001; d = 1.72). Early treatment before the age of 10 resulted in height gains of 13.92 cm (95 %CI 4.90-22.93; p = 0.006; d = 1.82) with estrogen and 6.8 cm (95 %CI 1.71-11.88; p = 0.010; d = 0.73) with testosterone.

CONCLUSIONS

Early intervention with low doses of steroids significantly reduced final height in individuals with tall stature, including those with MS, while also minimizing dose-dependent adverse effects.

摘要

目的

在门诊环境中评估性类固醇激素治疗对身材高大患者(包括诊断为马凡综合征(MS)的患者)15年以上最终身高的影响。

方法

这项回顾性队列研究回顾了因身材高大而转诊患者的病历。描述性统计描述样本特征,独立样本t检验和配对t检验评估最终身高(FH)和治疗开始时身高(HTS)的变化。单因素方差分析(ANOVA)评估治疗开始时的实足年龄、治疗开始时的骨龄和青春期阶段对FH和HTS的影响。

结果

共纳入55例身材高大的个体(51%为男性),其中35例(64%)临床确诊为MS。其中,34例(62%)接受了低剂量类固醇治疗。青春期前接受治疗的患者平均身高增加25.56 cm(95%CI 20.40 - 30.73;p < 0.001;d = 2.86),而青春期接受治疗的患者平均身高增加11.93 cm(95%CI 8.69 - 15.18;p < 0.001;d = 1.72)。10岁前早期治疗,使用雌激素身高增加13.92 cm(95%CI 4.90 - 22.93;p = 0.006;d = 1.82),使用睾酮身高增加6.8 cm(95%CI 1.71 - 11.88;p = 0.010;d = 0.73)。

结论

早期使用低剂量类固醇进行干预可显著降低身材高大个体(包括MS患者)的最终身高,同时也将剂量依赖性不良反应降至最低。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b869/12039514/7116183c0013/gr1.jpg

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